On January 13, 2021 Precigen, Inc. (Nasdaq: PGEN), a biopharmaceutical company specializing in the development of innovative gene and cell therapies to improve the lives of patients, reported that clinical and preclinical updates at the 39th Annual J.P. Morgan Healthcare Conference (Press release, Precigen, JAN 13, 2021, View Source [SID1234573979]). Helen Sabzevari, PhD, President and CEO of Precigen, presented a summary of 2020 clinical achievements and set forth Precigen’s clinical goals for 2021.

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In addition to reviewing the latest clinical updates for Precigen’s PRGN-3005 UltraCAR-T, PRGN-3006 UltraCAR-T, AG019 ActoBiotics, the presentation included the following additional announcements:

UltraCAR-T Library Approach: Precigen introduced the Company’s vision for a new UltraCAR-T library approach to transform the personalized cell therapy landscape for cancer patients. Precigen’s goal is to develop and validate a library of non-viral plasmids to target tumor-associated antigens. Enabled by design and manufacturing advantages of UltraCAR-T, coupled with the capabilities of the UltraPorator system, Precigen is working to empower cancer centers to deliver personalized, autologous UltraCAR-T treatment with overnight manufacturing to any cancer patient. Based on the patient’s cancer indication and biomarker profile, one or more non-viral plasmids would be selected from the library to build a personalized UltraCAR-T treatment. After initial treatment, this approach has the potential to allow for redosing of UltraCAR-T targeting the same or new tumor-associated antigen(s) based on the treatment response and the changes in antigen expression of the patient’s tumor. Precigen believes that the combination of the advanced UltraVector DNA construction platform and the ease of overnight manufacturing gives this library approach a proprietary advantage over traditional T-cell therapies.
PRGN-2009 AdenoVerse Immunotherapy in HPV-associated Cancers: For the first time, Precigen provided preliminary data for the ongoing Phase I/II study of the first-in-class PRGN-2009 AdenoVerse immunotherapy to treat HPV-positive (HPV+) solid tumors. The Phase I study is evaluating safety and response of PRGN-2009 alone (Arm A) and in combination with bintrafusp alfa, an investigational bifunctional fusion protein, (Arm B) in patients with HPV-associated cancers under a Cooperative Research and Development Agreement (CRADA) with the National Cancer Institute (NCI).
PRGN-2009 differentiation: PRGN-2009 leverages Precigen’s UltraVector and AdenoVerse platforms to optimize HPV 16/18 antigen design for delivery via a proprietary gorilla adenovector with a large genetic payload capacity and the ability for repeat administrations.
Preclinical activity: Preclinical studies show that PRGN-2009 treatment induced a strong HPV-specific immune response and anti-tumor response in a syngeneic mouse model of HPV+ cancer.
Addressable patient population: HPV infections account for 5% of all cancers globally1 and 690,000 new cancer cases are attributable to HPV infections per year.2
Enrollment: Enrollment in the Phase I monotherapy dose escalation arm (Arm A) is complete and enrollment in the Phase I combination arm (Arm B) has initiated.
Clinical activity: All patients (N=6) enrolled in the Phase I monotherapy arm (Arm A) have received multiple PRGN-2009 administrations to date and repeated administration of PRGN-2009 treatment has been well-tolerated with no dose-limiting toxicities.
Preliminary correlative analysis of peripheral blood mononuclear cells (PBMC) from patients treated at Dose Level 1 demonstrated an increase in HPV 16 and/or HPV 18-specific T-cell response post PRGN-2009 administration in 100% (3 out of 3) of patients; and
An increase in magnitude and breadth of immune response has been shown with respect to repeat administration of PRGN-2009.
Preclinical data for two new AdenoVerse immunotherapies:
PRGN-2012 AdenoVerse Immunotherapy in Recurrent Respiratory Papillomatosis (RRP): Precigen presented data from preclinical studies in which PRGN-2012 was shown to induce robust HPV 6 and HPV 11-specific T-cell response in RRP patient samples in vitro. Precigen recently announced that the US Food and Drug Administration (FDA) cleared the Investigational New Drug (IND) application to initiate the Phase I clinical trial of PRGN-2012 AdenoVerse immunotherapy in adult patients with recurrent respiratory papillomatosis (RRP).
PRGN-2013 AdenoVerse Immunotherapy in Hepatitis B Virus (HBV) Infection: Precigen shared preclinical data for PRGN-2013, which incorporates novel HBV antigen design in a proprietary gorilla adenovector, which showed that PRGN-2013 induces superior cytotoxic T-cell response against more HBV epitopes in mice than a competitor vaccine candidate and decreases plasma levels of HBsAg, the key marker of chronic HBV infection.
"In spite of the challenges presented by the COVID-19 pandemic, Precigen has accomplished all of the clinical milestones we set for ourselves in 2020," said Helen Sabzevari, PhD, President and CEO of Precigen, "I am proud of the team and collaborators for their commitment and execution during this challenging time which made possible the continued advancement of our differentiated therapeutics aimed at benefiting patients with severe unmet needs."

Precigen’s J.P. Morgan presentation is available on the Company website in the Events & Presentations section at investors.precigen.com/events-presentations.